BETHESDA, Md. — The Cystic Fibrosis Foundation is investing up to $9 million in additional funds in Arcturus Therapeutics to test an inhaled messenger RNA (mRNA) therapy that could treat the underlying cause of cystic fibrosis in all people living with the disease, including those with two copies of rare and nonsense mutations.
Arcturus is developing an mRNA therapy that is designed to deliver the mRNA template to the lung cells to produce functional cystic fibrosis transmembrane conductance regulator (CFTR) protein. The mRNA would be delivered to cells within a lipid nanoparticle, a chemically coated fat-like particle designed to help the mRNA enter cells more easily.
“Messenger RNA therapy offers a way to treat all people with CF, especially those who can’t tolerate or don’t respond to modulator treatment,” said JP Clancy, MD, senior vice president of clinical research at the Foundation. “Delivering the therapy to the correct cells is a significant challenge, and we are excited by the progress Arcturus is making in this area.”
Dosing has been completed in a Phase 1a clinical trial of Arcturus’ RNA therapy in healthy people in New Zealand, and a Phase 1b trial in people with CF in New Zealand has been approved. The Foundation’s funding will support this trial, which is planned for the fourth quarter of 2023. Arcturus also plans to use the funding for additional research prior to a planned Phase 2 trial in 2024.
The additional $9 million in funding combined with the $15.6 million already provided to Arcturus brings our total commitment to nearly $25 million. Our new investment comes from the $500 million Path to a Cure, an ambitious research initiative to accelerate treatments for everyone with CF and ultimately deliver a cure. The Foundation is funding research into mRNA therapies because of their potential to treat all people with CF.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.
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